Highlights From the ISTH 2020 Virtual Congress
Phase I/II Trial of SPK-8011: Stable and Durable FVIII Expression for > 2 Years With Significant ABR Improvements in Initial Dose Cohorts Following AAV-Mediated FVIII Gene Transfer for Hemophilia A
L. George1,2, E. Eyster3, M. Ragni4, S. Sullivan5, B. Samelson-Jones1,2, M. Evans3, A. MacDougall6, M. Curran6, S. Tompkins6, K. Wachtel6, D. Takefman6, K. Reape6, F. Mingozzi6, P. Monahan6, X. Anguela6, K. High6
1Children's Hospital of Philadelphia, Philadelphia, United States
2Perelman School of Medicine at the University of Pennsylvania, Philadelphia, United States
3Pennsylvania State University Milton S. Hershey Medical Center, Hershey, United States
4University of Pittsburgh Medical Center and Hemophilia Center of Western Pennsylvania, Department of Medicine, Pittsburgh, United States
5Mississippi Center for Advanced Medicine, Madison, United States
6Spark Therapeutics, Philadelphia, United States
L. George1,2, E. Eyster3, M. Ragni4, S. Sullivan5, B. Samelson-Jones1,2, M. Evans3, A. MacDougall6, M. Curran6, S. Tompkins6, K. Wachtel6, D. Takefman6, K. Reape6, F. Mingozzi6, P. Monahan6, X. Anguela6, K. High6
1Children's Hospital of Philadelphia, Philadelphia, United States
2Perelman School of Medicine at the University of Pennsylvania, Philadelphia, United States
3Pennsylvania State University Milton S. Hershey Medical Center, Hershey, United States
4University of Pittsburgh Medical Center and Hemophilia Center of Western Pennsylvania, Department of Medicine, Pittsburgh, United States
5Mississippi Center for Advanced Medicine, Madison, United States
6Spark Therapeutics, Philadelphia, United States
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